TRENTON, N.J. (AP) — Bayer HealthCare Pharmaceuticals said Tuesday that its experimental, genetically engineered treatment for the most common type of bleeding disorder hemophilia met its main goal of reducing treatment frequency in a late-stage patient study.
The medicine, called BAY 94-9027, helped prevent damaging internal bleeding in patients when given less frequently than standard treatments, which many patients need every other day. Bayer's treatment helps prevent bleeding episodes when infused — or given through a slow intravenous drip through a tube — as a preventive treatment twice per week, every five days and even every seven days. The medicine also stopped bleeding when it did occur.
The 134 patients in the study all have hemophilia A, the most common type. It affects about one in 5,000 newborn boys, for a total of about 20,000 American males.
The generally inherited disorder occurs when a type of protein needed to cause clotting, called Factor VIII, is missing or at inadequate levels. There is no cure, and without preventive treatment with clotting medicines, repeated episodes of uncontrolled bleeding in the joints and eyes cause pain, increasing disability and a shortened lifespan.
Bayer's drug is a genetically engineered version of the Factor VIII clotting protein, with an attachment site added, to which a protective molecule binds. This keeps the clotting protein active much longer as it circulates through the bloodstream, enabling patients to go longer between their pricey infusions.
Bayer is doing further studies on the medicine, including testing its safety in children and during major surgery, and the company plans a new study in patients not previously treated with similar anticlotting medicines.
Bayer HealthCare, based in Whippany, N.J., makes prescription drugs, veterinary medicines and consumer health products. It's part of part of German conglomerate Bayer AG, which also makes chemicals for farming and high-tech materials.